Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Dravet Syndrome

Dravet Syndrome Clinical Trial

Official title:

A Multicenter, Randomized, Double-blind, Placebo- Controlled, Interventional Study to Assess the Safety and Efficacy of Pharmaceutical Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Subjects With Inadequately Controlled Dravet Syndrome

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02318563
• Other study ID #: INS011-14-025
• Status: Withdrawn
• Phase: Phase 3
• First received: December 12, 2014
• Last updated: January 2, 2018
• Start date: December 30, 2017
• Est. completion date: June 17, 2018

Study information

• Verified date: January 2018
• Source: INSYS Therapeutics Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This Phase 3 study will enroll participants diagnosed with Dravet Syndrome (DS) who are still experiencing at least one tonic-clonic, clonic, and/or focal seizures with motor components (FSMC) per week, despite ongoing treatment with up to three antiepileptic drugs (AEDs), and meet the other inclusion/exclusion criteria.

Following a 28-day baseline period, participants will begin an 84-day treatment period. Participants will be assigned to receive twice-daily doses of placebo or cannabidiol oral solution at the highest dose determined to be safe in a previous trial.

Following study completion, all participants will be invited to receive Cannabidiol Oral Solution in an open label extension study (under a separate protocol).

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: June 17, 2018
• Est. primary completion date: June 17, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 30 Years

Eligibility

Inclusion Criteria:

• Meets protocol-specified criteria for qualification and contraception, including clinical diagnosis of refractory DS and onset of seizures according to protocol-specified criteria

• Is able to speak and understand the language in which the study is being conducted, is able to understand the procedures and study requirements and has voluntarily signed and dated an informed consent form approved by the Institutional Review Board before the conduct of any study procedure

• In the opinion of the Investigator, the subject and/or parent(s)/caregiver(s) are able to keep accurate seizure diaries and the participant is able to take study drug and comply with the protocol, including dosing, medications and diet

Exclusion Criteria:

• Medical history is outside protocol-specified parameters

• Clinically significant history of allergic reactions or significant sensitivities to cannabinoids or to any of the other ingredients in the study drug

• Inadequate supervision by parents or guardians

• History or current use of dietary supplements, drugs or over-the counter medications outside protocol-specified parameters

• Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise: 1) the safety or well-being of the participant or study staff; 2) the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding); 3) the analysis of results

Related Conditions & MeSH terms

• Dravet Syndrome
• Epilepsies, Myoclonic
• Syndrome

Intervention

Drug:
• Cannabidiol Oral Solution
An oral solution containing pharmaceutical grade cannabidiol (nonplant-based)
• Placebo Solution
A matching oral solution containing no cannabidiol

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
INSYS Therapeutics Inc

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change in the frequency of tonic-clonic, clonic, and focal seizures with motor components		Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary	Percent change from baseline in the severity of tonic-clonic, clonic, and focal seizures with motor components		Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary	Percent change from baseline in the duration of tonic-clonic, clonic, and focal seizures with motor components		Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary	Percent change from baseline in the frequency of all seizure activity independent of seizure type		Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary	Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Improvement (CGI-I)		Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary	Change from baseline in parent(s)/caregiver(s) Clinical Global Impressions of Severity (CGI-S)		Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary	Change from baseline in Investigator CGI-I		Data point for observation period to data point for treatment period Weeks 9 through 12
Secondary	Change from baseline in Investigator CGI-S		Data point for observation period to data point for treatment period Weeks 9 through 12